A Prospective, Multicentre, Non-Interventional Study Evaluating the Bleeding Incidence in Patients with Von Willebrand Disease Undergoing On-Demand Treatment (WIL-29)

Study type

• Study topic:
  Disease /health condition
• Study type:
  Non-interventional study
• Scope of the study:
  Drug utilisation

  Other
• If ‘other’, further details on the scope of the study:
  Characterization of bleeding events and treatment pattern
• Data collection methods:
  Primary data collection

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  This is a prospective, multicentre, international, non-controlled non-interventional study (NIS).

Study drug and medical condition

• Medical condition to be studied:
  Von Willebrand's disease

Population studied

• Short description of the study population:
  Patients with type 3, type 2 (except 2N), or severe type 1 VWD undergoing routine ondemand treatment with a VWF-containing product.
• Age groups:
  Children (2 to < 12 years)

  Adolescents (12 to < 18 years)

  Adults (18 to < 46 years)

  Adults (46 to < 65 years)

  Adults (65 to < 75 years)

  Adults (75 to < 85 years)

  Adults (85 years and over)
• Estimated number of subjects:
  56

Study design details

• Main study objective:
  The primary objective of this study is to characterise the bleeding and treatment pattern of patients with type 3, type 2 (except 2N), or severe type 1 VWD undergoing routine ondemand treatment with a VWF-containing product.
• Outcomes:
  total annualised bleeding rate (TABR), Spontaneous annualised bleeding rate (SABR), VWF-containing product consumption, proportion of succesfully treated bleeding Events. proportion of succesfully treated surgeries, QoL, Safety
• Data analysis plan:
  descriptive statistical methods.